Clinical Trial Details

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Currently there is no known effective treatment for recurrent or resistant neuroblastoma.
Fenretinide is an anticancer agent that may work differently than standard chemotherapy. It
may cause the buildup of wax-like substances in cancer cells called ceramides. In laboratory
studies, it was found that if too much ceramide builds up in the neuroblastoma cells, they
die.
Fenretinide has been given by mouth as a capsule to many people, including children. When
Fenretinide is given in capsules, very little of the drug is absorbed through the intestines
into the body. This means patients have to take many capsules of fenretinide by mouth
several times a day. In this study, a new oral preparation of fenretinide (called 4-HPR/LXS
oral powder) is being tested to see if more fenretinide can be absorbed into the body.
4-HPR/LXS oral powder has been tested previously in a limited number of both children and
adult cancer patients.
Ketoconazole, commonly used to treat fungus infections, can increase fenretinide levels in
the body by interfering with the body's ability to break down fenretinide. Ketoconazole will
be given at the same time as the fenretinide powder.
There is preclinical data that shows that combining fenretinide and vincristine prolonged
survival in animal models, therefore, it is hoped that giving the vincristine with
fenretinide will work better against the neuroblastoma that either drug given alone.
About 70 children with neuroblastoma have been treated with various versions of the
fenretinide powder to date, including about a dozen children that also took the fenretinide
powder with ketoconazole, and no toxicities have occurred that limited the dosage and no
serious or unexpected side effects occurred. However, vincristine has never been given with
fenretinide or fenretinide plus ketoconazole before. Vincristine has been been given before
with ketoconazole to both children and adults with neuroblastomas and other cancers.